- Exosome-Based Delivery of miR-124 in a Huntington’s Disease Model
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Soon-Tae Lee, Wooseok Im, Jae-Jun Ban, Mijung Lee, Keun-Hwa Jung, Sang Kun Lee, Kon Chu, Manho Kim
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J Mov Disord. 2017;10(1):45-52. Published online January 18, 2017
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DOI: https://doi.org/10.14802/jmd.16054
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Abstract
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- Objective
Huntington’s disease (HD) is a genetic neurodegenerative disease that is caused by abnormal CAG expansion. Altered microRNA (miRNA) expression also causes abnormal gene regulation in this neurodegenerative disease. The delivery of abnormally downregulated miRNAs might restore normal gene regulation and have a therapeutic effect.
Methods
We developed an exosome-based delivery method to treat this neurodegenerative disease. miR-124, one of the key miRNAs that is repressed in HD, was stably overexpressed in a stable cell line. Exosomes were then harvested from these cells using an optimized protocol. The exosomes (Exo-124) exhibited a high level of miR-124 expression and were taken up by recipient cells.
Results
When Exo-124 was injected into the striatum of R6/2 transgenic HD mice, expression of the target gene, RE1-Silencing Transcription Factor, was reduced. However, Exo-124 treatment did not produce significant behavioral improvement.
Conclusion
This study serves as a proof of concept for exosome-based delivery of miRNA in neurodegenerative diseases.
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Citations
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Zhuoyang Yu, Yan Teng, Jing Yang, Lu Yang Neural Regeneration Research.2024; 19(2): 282. CrossRef - A vivid outline demonstrating the benefits of exosome-mediated drug delivery in CNS-associated disease environments
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Francesco D'Egidio, Vanessa Castelli, Giorgia Lombardozzi, Fabrizio Ammannito, Annamaria Cimini, Michele d'Angelo Neural Regeneration Research.2024; 19(9): 1991. CrossRef - Extracellular vesicles in nanomedicine and regenerative medicine: A review over the last decade
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Shijie Li, Zhixin Lei, Taolei Sun Cell Biology and Toxicology.2023; 39(1): 53. CrossRef - Exosomes based strategies for brain drug delivery
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Oscar P. B. Wiklander, Meadhbh Á. Brennan, Jan Lötvall, Xandra O. Breakefield, Samir EL Andaloussi Science Translational Medicine.2019;[Epub] CrossRef - Exosome-mediated therapeutic delivery: A new horizon for human neurodegenerative disorders’ treatment (with a focus on siRNA delivery improvement)
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Xiaohuan Xia, Yi Wang, Yunlong Huang, Han Zhang, Hongfang Lu, Jialin C. Zheng Progress in Neurobiology.2019; 183: 101694. CrossRef - Role of Exosomes in Central Nervous System Diseases
Wanying Liu, Xiaodan Bai, Ao Zhang, Juanjuan Huang, Shixin Xu, Junping Zhang Frontiers in Molecular Neuroscience.2019;[Epub] CrossRef - miR-124 and Parkinson’s disease: A biomarker with therapeutic potential
Efthalia Angelopoulou, Yam Nath Paudel, Christina Piperi Pharmacological Research.2019; 150: 104515. CrossRef - The Potential Role of MicroRNA-124 in Cerebral Ischemia Injury
Xiaolu Liu, Zhitao Feng, Lipeng Du, Yaguang Huang, Jinwen Ge, Yihui Deng, Zhigang Mei International Journal of Molecular Sciences.2019; 21(1): 120. CrossRef - Epigenetic regulation of astrocyte function in neuroinflammation and neurodegeneration
Matthew Neal, Jason R. Richardson Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease.2018; 1864(2): 432. CrossRef - Potential of Extracellular Vesicles in Neurodegenerative Diseases: Diagnostic and Therapeutic Indications
Mehrnaz Izadpanah, Arshia Seddigh, Somayeh Ebrahimi Barough, Seyed Abolhassan Shahzadeh Fazeli, Jafar Ai Journal of Molecular Neuroscience.2018; 66(2): 172. CrossRef - Regenerative Approaches in Huntington’s Disease: From Mechanistic Insights to Therapeutic Protocols
Jenny Sassone, Elsa Papadimitriou, Dimitra Thomaidou Frontiers in Neuroscience.2018;[Epub] CrossRef - Exosome-based small RNA delivery: progress and prospects
Mei Lu, Haonan Xing, Zhe Xun, Tianzhi Yang, Pingtian Ding, Cuifang Cai, Dongkai Wang, Xiaoyun Zhao Asian Journal of Pharmaceutical Sciences.2017;[Epub] CrossRef - Extracellular Vesicles in Brain Tumors and Neurodegenerative Diseases
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- Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders
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Wooseok Im, Jangsup Moon, Manho Kim
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J Mov Disord. 2016;9(3):136-143. Published online September 21, 2016
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DOI: https://doi.org/10.14802/jmd.16029
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Abstract
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- Gene therapy is a potential therapeutic strategy for treating hereditary movement disorders, including hereditary ataxia, dystonia, Huntington’s disease, and Parkinson’s disease. Genome editing is a type of genetic engineering in which DNA is inserted, deleted or replaced in the genome using modified nucleases. Recently, clustered regularly interspaced short palindromic repeat/CRISPR associated protein 9 (CRISPR/Cas9) has been used as an essential tool in biotechnology. Cas9 is an RNA-guided DNA endonuclease enzyme that was originally associated with the adaptive immune system of Streptococcus pyogenes and is now being utilized as a genome editing tool to induce double strand breaks in DNA. CRISPR/Cas9 has advantages in terms of clinical applicability over other genome editing technologies such as zinc-finger nucleases and transcription activator-like effector nucleases because of easy in vivo delivery. Here, we review and discuss the applicability of CRISPR/Cas9 to preclinical studies or gene therapy in hereditary movement disorders.
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- Cell Therapy Strategies vs. Paracrine Effect in Huntington’s Disease
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Wooseok Im, Manho Kim
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J Mov Disord. 2014;7(1):1-6. Published online April 30, 2014
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DOI: https://doi.org/10.14802/jmd.14001
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Abstract
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- Huntington’s disease (HD) is a genetic neurodegenerative disorder. The most common symptom of HD is abnormal involuntary writhing movements, called chorea. Antipsychotics and tetrabenazine are used to alleviate the signs and symptoms of HD. Stem cells have been investigated for use in neurodegenerative disorders to develop cell therapy strategies. Recent evidence indicates that the beneficial effects of stem cell therapies are actually mediated by secretory molecules, as well as cell replacement. Although stem cell studies show that cell transplantation provides cellular improvement around lesions in in vivo models, further work is required to elucidate some issues before the clinical application of stem cells. These issues include the precise mechanism of action, delivery method, toxicity and safety. With a focus on HD, this review summarizes cell therapy strategies and the paracrine effect of stem cells.
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